A Study to Assess Safety and Efficacy of KarXT in Adult Patients With Schizophrenia

NCT ID: NCT03697252

Last Updated: 2020-10-26

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

182 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-09-18

Study Completion Date

2019-09-04

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is a Phase 2, randomized, double-blinded, placebo-controlled, inpatient study to examine the efficacy, safety, and tolerability profile of KarXT in adult subjects diagnosed with DSM-5 schizophrenia who are in an acute exacerbation phase. The primary objective of the study is to assess the efficacy of KarXT (a fixed combination of xanomeline and trospium chloride) (xanomeline 125 mg/trospium 30 mg twice daily \[BID\]) versus placebo in reducing Positive and Negative Syndrome Scale (PANSS) total scores in adult inpatients with a Diagnostic and Statistical Manual-Fifth Edition (DSM-5) diagnosis of schizophrenia. The secondary objectives of the study are to assess overall safety and tolerability of KarXT in adult inpatients with a DSM-5 diagnosis of schizophrenia.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Schizophrenia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

KarXT

Group Type EXPERIMENTAL

Xanomeline and Trospium Chloride Capsules

Intervention Type DRUG

Xanomeline 50 mg/trospium 20 mg BID on days 1-2 followed by xanomeline 100 mg/trospium 20 mg BID on days 3-7. The dose is increased to xanomeline 125 mg/trospium 30 mg BID on days 8-34 unless the subject is experiencing adverse events from the xanomeline 100 mg/trospium 20 mg dose. Subjects who were increased to xanomeline 125 mg/trospium 30 mg will have the option to return to xanomeline 100 mg/trospium 20 mg depending on clinical response and tolerability. Dosing must not change after Visit 7 of the study (at 21 ± 2 days of dosing) and may be decreased for tolerability reasons no more than once during the study.

Placebo

Group Type PLACEBO_COMPARATOR

Placebo Capsules

Intervention Type DRUG

Placebo Capsules

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Xanomeline and Trospium Chloride Capsules

Xanomeline 50 mg/trospium 20 mg BID on days 1-2 followed by xanomeline 100 mg/trospium 20 mg BID on days 3-7. The dose is increased to xanomeline 125 mg/trospium 30 mg BID on days 8-34 unless the subject is experiencing adverse events from the xanomeline 100 mg/trospium 20 mg dose. Subjects who were increased to xanomeline 125 mg/trospium 30 mg will have the option to return to xanomeline 100 mg/trospium 20 mg depending on clinical response and tolerability. Dosing must not change after Visit 7 of the study (at 21 ± 2 days of dosing) and may be decreased for tolerability reasons no more than once during the study.

Intervention Type DRUG

Placebo Capsules

Placebo Capsules

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

KarXT

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Subject is aged 18-60 years, inclusive, at screening
2. Subject has a primary diagnosis of schizophrenia established by a comprehensive psychiatric evaluation based on the DSM-5 (American Psychiatric Association 2013) criteria and confirmed by Mini International Neuropsychiatric Interview for Schizophrenia and Psychotic Disorder Studies (MINI) version 7.0.2.
3. Subject is experiencing an acute exacerbation or relapse of symptoms, with onset less than 2 months before screening
4. Positive and Negative Syndrome Scale total score between 80 and 120, inclusive, at screening

1. Score of ≥ 4 (moderate or greater) for ≥ 2 of the following Positive Scale (P) items at screening:
2. Item 1 (P1; delusions)
3. Item 2 (P2; conceptual disorganization)
4. Item 3 (P3; hallucinatory behavior)
5. Item 6 (P6; suspiciousness/persecution)
5. There should not be a change (improvement) in PANSS total score between screening and baseline of more than 20%
6. Subjects taking a depot antipsychotic could not have received a dose of medication for at least 1 and a half injection cycles before baseline (eg, 3 or more weeks off for a 2-week cycle)
7. Subject is capable of providing informed consent

1. A signed ICF must be provided before any study assessments are performed
2. Subject must be fluent (oral and written) in English in order to consent
8. Subject must have CGI-S score of ≥ 4 at screening and baseline visits
9. Body mass index must be ≥ 18 and ≤ 40 kg/m2
10. Both females of child bearing potential and males with partners of child bearing potential must be willing to use a double-barrier method of birth control (ie, any double combination of male or female condom with spermicidal gel, diaphragm, sponge, or cervical cap with spermicidal gel) during the study and for 7 days after the last dose of study drug.
11. Subject has an identified reliable informant

Exclusion Criteria

1. Any primary DSM-5 disorder other than schizophrenia within 12 months before screening (confirmed using MINI version 7.0.2 at screening)
2. History or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, or oncologic disease or any other condition that, in the opinion of the investigator, would jeopardize the safety of the subject or the validity of the study results, to exclude patients with human immunodeficiency virus (HIV), cirrhosis, biliary duct abnormalities, hepatobiliary carcinoma, and/or active hepatic viral infections based on the liver function test results.
3. History of or high risk of urinary retention, gastric retention, or narrow-angle glaucoma
4. History of irritable bowel syndrome (with or without constipation) or serious constipation requiring treatment within the last 6 months
5. Has a DSM-5 diagnosis of moderate to severe substance abuse disorder (except tobacco use disorder) within the 12 months before screening (confirmed using MINI version 7.0.2 at screening), or current abuse as determined by urine toxicology screen or alcohol test. A screening subject with mild substance abuse disorder within the 12 months before screening must be discussed and agreed upon with the medical monitor before he/she can be allowed into the study.
6. Clinically significant abnormal finding on the physical examination, medical history, ECG, or clinical laboratory results at screening
7. Pregnant, lactating, or less than 3 months postpartum. Sperm donation is not allowed for 90 days after the final dose of study drug
8. If, in the opinion of the investigator (and/or Sponsor), subject is unsuitable for enrollment in the study or subject has any finding that, in the view of the investigator (and/or Sponsor), may compromise the safety of the subject or affect their ability to adhere to the protocol visit schedule or fulfill visit requirements
9. Subject has had psychiatric hospitalization(s) for more than 30 days (cumulative) during the 90 days before screening
10. Subject has a history of treatment resistance to schizophrenia medications defined as failure to respond to 2 adequate courses of pharmacotherapy (a minimum of 4 weeks at an adequate dose per the label) or required clozapine within the last 12 months
11. Risk of violent or destructive behavior
12. Current involuntary hospitalization or incarceration
13. Participation in another clinical study in which the subject received an experimental or investigational drug agent within 3 months of screening
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Karuna Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Stephen Brannan, MD

Role: STUDY_DIRECTOR

Karuna Therapeutics

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Woodland International Research Group, LLC

Little Rock, Arkansas, United States

Site Status

Synergy East

Lemon Grove, California, United States

Site Status

Collaborative Neuroscience Network, LLC.

Long Beach, California, United States

Site Status

NRC Research Institute

Orange, California, United States

Site Status

Artemis Institute for Clinical Research

San Diego, California, United States

Site Status

Atlanta Center for Medical Research

Atlanta, Georgia, United States

Site Status

CBH Health, LLC

Gaithersburg, Maryland, United States

Site Status

Hassman Research Institute

Berlin, New Jersey, United States

Site Status

Midwest Clinical Research Center (and IP Shipment)

Dayton, Ohio, United States

Site Status

Community Clinical Research, Inc.

Austin, Texas, United States

Site Status

InSite Clinical Research, LLC

DeSoto, Texas, United States

Site Status

Pillar Clinical Research, LLC

Richardson, Texas, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Yeung PP, Breier A, Zhu H, Kaul I, Marcus RN. Xanomeline and Trospium Chloride for the Treatment of Agitation Associated With Schizophrenia: PANSS-Excited Component Results From 3 Randomized, Double-Blind, Placebo-Controlled EMERGENT Trials. J Clin Psychiatry. 2025 Mar 24;86(2):24m15668. doi: 10.4088/JCP.24m15668.

Reference Type DERIVED
PMID: 40215379 (View on PubMed)

Citrome L, Neugebauer NM, Meli AA, Kando J. Xanomeline and Trospium Chloride Versus Placebo for the Treatment of Schizophrenia: A Post Hoc Analysis of Number Needed to Treat, Number Needed to Harm, and Likelihood to Be Helped or Harmed. Neuropsychiatr Dis Treat. 2025 Apr 5;21:761-773. doi: 10.2147/NDT.S503494. eCollection 2025.

Reference Type DERIVED
PMID: 40212458 (View on PubMed)

Kaul I, Claxton A, Sawchak S, Sauder C, Brannan SK, Raj E, Ruan S, Konis G, Brown D, Cutler AJ, Marcus R. Safety and Tolerability of Xanomeline and Trospium Chloride in Schizophrenia: Pooled Results From the 5-Week, Randomized, Double-Blind, Placebo-Controlled EMERGENT Trials. J Clin Psychiatry. 2025 Feb 26;86(1):24m15497. doi: 10.4088/JCP.24m15497.

Reference Type DERIVED
PMID: 40047530 (View on PubMed)

Sauder C, Allen LA, Baker E, Miller AC, Paul SM, Brannan SK. Effectiveness of KarXT (xanomeline-trospium) for cognitive impairment in schizophrenia: post hoc analyses from a randomised, double-blind, placebo-controlled phase 2 study. Transl Psychiatry. 2022 Nov 21;12(1):491. doi: 10.1038/s41398-022-02254-9.

Reference Type DERIVED
PMID: 36414626 (View on PubMed)

Weiden PJ, Breier A, Kavanagh S, Miller AC, Brannan SK, Paul SM. Antipsychotic Efficacy of KarXT (Xanomeline-Trospium): Post Hoc Analysis of Positive and Negative Syndrome Scale Categorical Response Rates, Time Course of Response, and Symptom Domains of Response in a Phase 2 Study. J Clin Psychiatry. 2022 May 11;83(3):21m14316. doi: 10.4088/JCP.21m14316.

Reference Type DERIVED
PMID: 35552528 (View on PubMed)

Targum SD, Murphy C, Breier A, Brannan SK. Site-independent confirmation of primary site-based PANSS ratings in a schizophrenia trial. J Psychiatr Res. 2021 Dec;144:241-246. doi: 10.1016/j.jpsychires.2021.10.027. Epub 2021 Oct 21.

Reference Type DERIVED
PMID: 34700212 (View on PubMed)

Brannan SK, Sawchak S, Miller AC, Lieberman JA, Paul SM, Breier A. Muscarinic Cholinergic Receptor Agonist and Peripheral Antagonist for Schizophrenia. N Engl J Med. 2021 Feb 25;384(8):717-726. doi: 10.1056/NEJMoa2017015.

Reference Type DERIVED
PMID: 33626254 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

KAR-004

Identifier Type: -

Identifier Source: org_study_id